How do I Choose the Best Duchenne Muscular Dystrophy Treatment?

Duchenne muscular dystrophy is an inherited type of muscular dystrophy that progresses much more rapidly than other types. There is no cure for Duchenne muscular dystrophy, but treatment typically is a combination of physical therapies, lifestyle changes and medications. The best Duchenne muscular dystrophy treatment will take into account the individual’s particular symptoms and disease progression and will be a multifaceted approach. This typically will involve different specialists working together to create a support network for a patient. You can choose the best Duchenne muscular dystrophy treatment by researching all of the options thoroughly and consulting with a medical professional.

One type of Duchenne muscular dystrophy treatment that you could choose is pharmaceutical treatment. Muscle strength and muscle function are affected by the disease, so people who have Duchenne muscular dystrophy will often feel muscle stiffness and weakness. Medications such as phenytoin or dantrolene can help with these symptoms. An anti-inflammatory corticosteroid medication called prednisone is sometimes prescribed to treat muscle deterioration.

Physical therapy is a very important facet of Duchenne muscular dystrophy treatment. As muscles deteriorate and become weaker, painful contractures may develop. Contractures happen when the muscles weaken and the tendons shorten, making joints stiffer and more difficult to move. Physical therapy can help a person with muscular dystrophy to continue to move normally and reduce pain. Hot baths are another way to reduce pain and improve joint motion.

Orthopedic appliances are another Duchenne muscular dystrophy treatment that you might choose. Some types of orthopedic appliances, such as braces, can help support weakened muscles. Canes and walkers can enable a person to be more mobile, allowing him or her to retain independence as the disease progresses. A ventilator can compensate for weakened respiratory muscles.

Some contractures become so severe that they require surgery to treat. Surgery can lengthen tendons, increasing the range of movement and reducing pain. Typically if a contracture does not respond to physical therapy over a period of time, a physician will consider surgical treatment. Surgery also can be an effective way to correct for the spine curvature that can develop as the disease progresses.

A newer type of Duchenne muscular dystrophy treatment, called gene therapy, might be an effective treatment to consider. One of the causes of Duchenne muscular dystrophy is a lack of dystrophin, a protein that plays a role in muscle maintenance. Scientists have created a gene that produces small amounts of dystrophin. In a laboratory test where this gene was given to mice, the mice produced more dystrophin in the targeted muscles. A year later, the therapy was still effective.

Gene therapy also has been effective when tested in isolated human cells. Small clinical trials using gene therapy as a Duchenne muscular dystrophy treatment have had some success. Gene therapy could be the best treatment for Duchenne muscular dystrophy as the technology progresses.